Leading researchers outline principles for conduct of comparative effectiveness research

Wednesday, September 12, 2012

A new set of principles developed by a group of leading researchers could help to ensure more consistency in how comparative effectiveness research (CER) is planned and conducted. These guiding principles were published in the September issue of The Journal of Comparative Effectiveness Research.

“These are voluntary principles that can help to improve the quality of CER, which in turn can help patients and providers make better treatment decisions,” said Bryan R. Luce, PhD, MBA, United BioSource and the University of Washington, and one of the researchers who developed the guidelines. “For CER to succeed in guiding health decisions, it should be planned and conducted with rigor and transparency.”

To arrive at these best research practice principles, the researchers examined existing health technology assessment principles and engaged multiple CER experts and stakeholders for feedback.

The resulting 13 principles include ensuring that research objectives are clear, being transparent in how the research is conducted, involving stakeholders, including especially relevant decision makers, in a meaningful way throughout the research process, considering perspectives and interests from a wide range of stakeholders, using relevant comparators, and evaluating relevant outcomes and the impact of individual treatment effects on patients. The researchers acknowledge that “no one study will necessarily be able to fully meet every principle to the letter,” but that CER should always endeavor to fulfill, and never ignore, their intent.

“Going forward, it will be important to evaluate how well CER studies adhere to these principles. We can then determine whether CER studies that use those principles are having an impact on patient care or are being appropriately adopted into guidelines and clinical practice,” said Robert W. Dubois, MD, PhD, National Pharmaceutical Council, and one of the researchers who developed the guidelines.

The principles and paper were developed by Luce; Dubois; Michael F. Drummond, PhD, University of York; Peter J. Neumann, ScD, Tufts Medical Center and Tufts University School of Medicine; Bengt Jönsson, PhD, Stockholm School of Economics (Sweden); Uwe Siebert, MD, MPH, MSc, ScD, University of Health Sciences, Medical Informatics and Technology (Austria) and Harvard School of Public Health; and J. Sanford Schwartz, MD, Wharton School of Business and Perelman School of Medicine, University of Pennsylvania.